It is hoped that with the cooperation of this organization, we will take steps towards the awareness and treatment of such rare diseases and patients.
Traditional CBT as effective as exposure therapy for fibromyalgia
There does not appear to be any profound differences between so-called exposure-based CBT and traditional CBT in the treatment of fibromyalgia, according to a study led by researchers at Karolinska Institutet. Both forms of treatment produced a significant reduction in symptoms in people affected by the disease. The study is one of the largest to date to compare different treatment options for fibromyalgia and is published in the journal PAIN.
About 200,000 people in Sweden currently live with fibromyalgia, a long-term pain syndrome that causes great suffering for patients through widespread pain, fatigue, and stiffness in the body. There is no cure for fibromyalgia. Existing drugs often have insufficient effect, raising the need for more effective treatment methods. Cognitive behavioral therapy (CBT) has shown some effect, but there is a lack of trained CBT practitioners. There is also a lack of knowledge about which form of CBT is most effective.
The study compared two different forms of internet-delivered cognitive behavioral therapy in terms of how well they reduce the symptoms and functional impact of fibromyalgia.
In brief, exposure-based CBT involves the participant systematically and repeatedly approaching situations, activities, and stimuli that the patient has previously avoided because the experiences are associated with pain, psychological discomfort, or symptoms such as fatigue and cognitive problems.
In traditional CBT, the participant is presented with several different strategies to work on during treatment, such as relaxation, activity planning, physical exercise, or strategies for managing negative thoughts and improving sleep.
The study showed that traditional CBT was by and large equivalent to the newer treatment form of exposure-based CBT.
The randomized study involved 274 people with fibromyalgia, who were randomly assigned to be treated with traditional or exposure-based CBT. The treatments were delivered entirely online and all participants had regular contact with their therapist.
Participants answered questions about their mood and symptoms before, during, and after treatment. After the 10-week treatment, 60 percent of those who received exposure-based CBT and 59 percent of those who received traditional CBT reported that their treatment had helped them.
“The fact that both treatments were associated with a significant reduction in the participants’ symptoms and functional impairment and that the effects were sustained for 12 months after completion of the treatment, indicates that the internet as a treatment format can be of great clinical benefit for people with fibromyalgia,” says Maria Hedman-Lagerlöf. “This is good news because it enables more people to access treatment.”
The study is the second largest to compare different psychological treatment options for fibromyalgia, according to the researchers.
“Our study is also one of the first to compare with another active, established psychological treatment,” says Maria Hedman-Lagerlöf.
The study was a collaboration between Karolinska Institutet and Uppsala University. It was financed by The Bank of Sweden Tercentenary Foundation and none of the researchers have declared any conflicts of interest.
UB research identifies key mechanism in infantile cystinosis
University at Buffalo research has identified how a misstep in the genesis of a key component of the kidney causes infantile cystinosis, a rare disease that significantly shortens the lifespan of patients. Published Nov. 30 in the International Journal of Molecular Sciences, the work reveals that the mechanisms that cause the disease could be addressed and potentially cured through the genome-editing technique CRISPR. That could make kidney transplants, the most effective treatment currently available for these patients, unnecessary.
Infantile cystinosis, the most common and most severe type of cystinosis, occurs as the result of an accumulation in the body’s cells of cystine, an amino acid. The buildup damages cells throughout the body, especially the kidneys and the eyes. Treatment consists of medications that work to lower the level of cystine in the body, as well as therapies that address the impaired growth of these children due to the inability to properly absorb nutrients. Some children require feeding tubes. Eventually, patients with infantile cystinosis, also called nephropathic cystinosis, will require dialysis and a kidney transplant.
Promise of stem cells
Human-induced pluripotent stem cells (hiPSCs) are stem cells that can differentiate into many different cell types. They hold tremendous potential for studying genetic diseases; the drawback has been that differentiation into certain cell types has been problematic. Such is the case with many cell types found in the kidney.
But a new protocol developed by this research team was successful.
Ramkumar Thiyagarajan, PhD, assistant professor of geriatric studies at the University of Kansas and formerly a postdoctoral fellow at UB, is first author on the paper.
The protocol involved extracting stem cells from a healthy individual and an individual with infantile cystinosis. The researchers developed a culture medium to grow stem cells that included a small number of defined components present in blood, including insulin, specific proteins, growth factors and others. “Conducting the differentiation protocol under these conditions occurred in a timely manner,” says Taub, “we didn’t have to wait for weeks on end, and it occurred in a reproducible manner.”
The researchers were able to efficiently differentiate the hiPSCs into the kidney proximal tubule, the type of nephron in the kidney that is impaired in infantile cystinosis, as well as in other kidney diseases.
“Unlike in other studies, we were able to retain a number of markers in the tubule that are physiologically important in the kidney’s reabsorptive functions,” says Taub. “Although these markers were expressed in both the normal and the cystinosis-derived hiPSCs, the genesis of the tubule was impaired in the cystinosis-derived cells, mimicking what happens in infantile cystinosis.”
A potential cure
That finding means that the CRISPR genome-editing technique could be used to repair the defective genome and potentially cure the disease. “The normal gene can be introduced in the genome of cystinotic hiPSCs, which can then be injected in the kidney to replace the defective proximal tubules of individuals with infantile cystinosis,” Taub says.
“In cystinotic individuals, it is the renal proximal tubule that degenerates, presumably due to programmed cell death,” explains Taub, “so the entire kidney would not need to be replaced. The defective renal proximal tubules of individuals with this disease can be replaced with normal tubules following the introduction of the normal gene into cystinotic hiPSCs obtained from the patient. And because these tubules are from cells derived from the patient, there should be no problem with tissue rejection.”
The findings are applicable to other kidney diseases where the renal proximal tubule is damaged, including acute kidney injury that can lead to chronic kidney disease and renal failure, and can be fatal.
Initial studies will need to be conducted with animal models as well as with in vitro tissue culture cells.
The research was funded by UB’s WNYSTEM and The Cystinosis Research Foundation.
Source:
Journal reference:
Thiyagarajan, R & Taub, M. (2023). Studies with Human-Induced Pluripotent Stem Cells Reveal That CTNS Mutations Can Alter Renal Proximal Tubule Differentiation. International Journal of Molecular Sciences.
WPA Asia-Pacific Regional Meeting
4 – 5 November, Dubai, UAE
with attendance of Dr yaser davoudian
The vibrant city of Dubai hosted the highly anticipated Asia-Pacific Regional Meeting of the World Patient Alliance (WPA) on November 4-5, 2023. The gathering brought together global leaders from patient organizations, passionate patient advocates, and dedicated healthcare stakeholders.
A dynamic combination of face-to-face and virtual interaction: One of the outstanding features of the Asia-Pacific Regional Meeting was its hybrid format. This forward-thinking approach allowed participants to connect both in person and virtually, ensuring a diverse and inclusive experience that transcended geographic boundaries. This reflects WPA’s commitment to creating meaningful engagement for a global audience.
Insights from keynote speakers: The event featured a mix of keynote speakers from national and international health organizations, each bringing their unique perspectives to the fore. Insights shared by Mondher Letaief, Shin Ushiro, Lamya Alhazani, Candace Henley, Christiane El Ferzli, Nicole Sheahan, Kimberly Galia, and in-house speakers Andrew Spiegel Esq., Penney Cowan, and Hussain Jafri added depth to the discussions. Overview of key topics: The diverse range of topics covered at the Asia Pacific Regional Meeting provides a comprehensive look at the challenges and opportunities in patient advocacy. From “The Art of Patient Advocacy” to discussions on “Universal Health Coverage” and surveying “Health Emergencies in the Region”, each session contributed to a comprehensive understanding of the current healthcare landscape.
Panel Discussions: Expert Conversation: The event agenda was enriched with panel discussions featuring healthcare experts and patient safety champions. Anas Nofil, Ken Tanda, Edmond Love, Arij Saqer, JS Arora, Mekia Majrashi, Inge Damanti and Regina Kamuga shared their experiences and perspectives, fueling a rich conversation about the future of patient safety and healthcare. Leadership Insights: WPA leadership, including Andrew Spiegel, Penny Cowan, and Hussain Jafari, shared their insights on the success of the event. Their insights highlighted the growing role of patient advocacy in a changing world and expressed appreciation for the global community’s commitment to patient-centered solutions.
A celebration of collaboration and shared dedication: More than just a meeting, the Asia-Pacific Regional Gathering was a celebration of collaboration, dialogue and a shared commitment to improving healthcare for patients worldwide. It provided a fertile ground for making connections, sharing best practices, and exploring solutions that resonate across borders.
The event aims to foster a global dialogue on patient safety, quality healthcare and advocacy. We believe that our active participation will also contribute greatly to your own understanding of the critical issues surrounding patient engagement in healthcare.
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